The United States has imposed significant restrictions on the gene therapy treatment Elevidys, used for Duchenne muscular dystrophy, following the deaths of two adolescent patients. The decision, announced by the Food and Drug Administration (FDA) on July 7, 2025, will limit the therapy’s use to individuals who are able to walk and are older than four years. This measure comes in response to alarming reports of acute liver failure among non-ambulatory patients who received the treatment.
Both patients who suffered fatal outcomes were unable to walk at the time they began the therapy. Duchenne muscular dystrophy is a rare and progressive condition that primarily affects boys, leading to severe muscle deterioration and generally resulting in a life expectancy of around 28 years. The FDA’s new warning label for Elevidys marks a critical shift in its approval status, emphasizing the need for increased safety monitoring.
In the summer of 2025, Sarepta Therapeutics, the biopharmaceutical company behind Elevidys, temporarily halted distribution to non-ambulatory patients. This decision followed the FDA’s suspension of clinical trials and the initiation of a comprehensive safety review. The FDA requested Sarepta to cease all shipments of the therapy, further highlighting the urgency of the situation.
Regulatory Response and Safety Measures
The FDA’s new guidelines recommend that patients receiving Elevidys undergo weekly liver function monitoring for the first three months after treatment begins. This precaution aims to mitigate risks associated with potential liver complications linked to the therapy. The agency’s actions reflect increasing concerns about the safety and efficacy of treatments for Duchenne muscular dystrophy.
In July 2025, Europe’s Committee for Medicinal Products for Human Use also expressed reservations about Elevidys, advising against its marketing approval. The committee cited insufficient data to convincingly demonstrate the treatment’s effectiveness in improving movement abilities among patients.
The debate surrounding Elevidys has sparked tension within the FDA, as advocacy groups and affected families continue to seek solutions for a condition that has limited treatment options. The push for access to effective therapies for Duchenne muscular dystrophy remains a critical issue, with many stakeholders advocating for further research and development in this area.
While the FDA’s actions aim to prioritize patient safety, the limitations placed on Elevidys may leave many families facing difficult choices. As the biopharmaceutical landscape evolves, the quest for viable treatments for Duchenne muscular dystrophy continues to be a complex and pressing challenge.
